Rare Disease Day, held annually on 28th February, raises awareness of people living with rare diseases across the world, their families, and their carers. The key aims of the campaign are to achieve equitable access to diagnosis, treatment, and social opportunity for those affected by rare diseases. 28 February 2025
Privately-held Austria firm pharmaand GmbH (pharma&) today announced that the National Institute of Health and Care Excellence (NICE) has issued a positive recommendation for its Rubraca (rucaparib). 21 February 2025
UK health technology assessor the National Institute for Health and Care Excellence (NICE) has issued Final Draft Guidance recommending the use Breyanzi (lisocabtagene maraleucel) for National Health Service (NHS) in England. 21 February 2025
In a U-turn for the UK health technology assessor, the National Institute for Health and Care Excellence (NICE) has today approved Fintepla (fenfluramine) for the treatment of Lennox-Gastaut syndrome (LGS) in patients from two years of age. 20 February 2025
A new report from industry trade group the Pharmaceutical Research and Manufacturers of America (PhRMA) has shed a new light on the flow of dollars through the pharmaceutical supply chain for commercially insured patients taking retail medicines. 14 February 2025
Antengene has announced that its multiple myeloma treatment, Xpovio (selinexor), will be included in Taiwan’s National Health Insurance (NHI) reimbursement scheme. 13 February 2025
Santhera Pharmaceuticals, a Swiss specialty pharma focused on rare diseases, has announced an agreement with the German National Association of Statutory Health Insurance Funds (GKV-SV) on the reimbursement for Agamreee (vamorolone) to treat Duchenne muscular dystrophy (DMD). 13 February 2025
Australian radiopharma company Telix Pharmaceuticals has gained approval from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for its prostate cancer imaging agent, Illuccix (gallium-68 gozetotide). 13 February 2025
The USA-based Institute for Clinical and Economic Review (ICER), England’s National Institute for Health and Care Excellence (NICE), and Canada’s Drug Agency (CDA-AMC) have formed the Health Economics Methods Advisory (HEMA) initiative. The goal of HEMA is to critically and independently research some of the most pressing topics in global health economics and health technology assessment (HTA) methods. 12 February 2025
On Sunday night, one of the USA’s largest compounders, Hims & Hers, ran a multi-million dollar Super Bowl advert about its provision of GLP-1 weight loss medications. 10 February 2025
US health technology assessor the Institute for Clinical and Economic Review (ICER) yesterday posted its revised Evidence Report assessing the comparative clinical effectiveness and value of suzetrigine, from Vertex Pharmaceuticals, for the treatment of acute pain. 6 February 2025
At Sweden’s annual life science conference, stakeholders from healthcare, academia, and industry discussed implementing the country’s new life science strategy. 3 February 2025
Shares of US drugmaker Vertex Pharmaceuticals jumped 8.4% to $475.00 in after-hours trading yesterday after it announced that the US Food and Drug Administration (FDA) has approved Journavx (suzetrigine), an oral, non-opioid, highly selective NaV1.8 pain signal inhibitor for the treatment of adults with moderate-to-severe acute pain. 31 January 2025
New US President Donald Trump and his administration have confirmed that lowering the cost of prescription drugs for Americans is a top priority, suggesting that they will continue the work of the previous regime. 30 January 2025
UK health technology assessor the National Institute for Health and Care Excellence (NICE) has recommended Acarizax (12 SQ-HDM SLIT, also known as ‘sublingual immunotherapy’) for the treatment of moderate to severe house dust mite allergic rhinitis disease in people 12 to 65 years that is persistent despite symptom-relieving medicine. 30 January 2025
The headline payment percentage for newer medicines under the Voluntary Scheme for Branded Medicines Pricing and Access (VPAG) has been set at 22.9% for 2025, raising concerns from the UK pharma industry. 28 January 2025
Patients For Affordable Drugs, Public Citizen, Social Security Works and other campaigners are to gather in Washington, DC next week with the aim of delivering a message to pharma. 23 January 2025
A controversial proposal to change the Inflation Reduction Act’s pricing framework for rare disease drugs has been dropped from the Senate’s reconciliation package, marking a win for patient advocacy groups that lobbied against it. The ORPHAN Cures Act, which sought to shield some rare disease drugs from Medicare price talks, was notably absent from the text released this week by the Senate Finance Committee. 18 June 2025
US trade association PhRMA (Pharmaceutical Research and Manufacturers of America) has cited a new IQVIA study that found that Medicare Part D plans routinely deny access to prescribed medicines for patients with newly-diagnosed chronic conditions. 17 June 2025
The National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending GSK’s Blenrep (belantamab mafodotin) in combination with bortezomib and dexamethasone (BVd) for second-line multiple myeloma treatment of adults who are refractory to or intolerant of lenalidomide. 13 June 2025
Africa is grappling with a worsening mpox crisis, with a critical shortfall of vaccine doses threatening to derail containment efforts. Public health leaders are warning that the scarcity is exacerbating the spread of the virus, even as the World Health Organization (WHO) maintains that mpox remains a public health emergency of international concern. 10 June 2025
A sweeping legislative package backed by US President Donald Trump could strip health insurance from more than 16 million Americans over the next decade, according to a new analysis from the Congressional Budget Office. 5 June 2025
Sogroya (somapacitan injection) has been recommended by UK health technology assessor the National Institute for Health and Care Excellence (NICE) as a treatment for people aged 3 to 17 years with pediatric growth hormone deficiency, the UK subsidiary of Denmark’s Novo Nordisk revealed yesterday. 4 June 2025
The UK risks losing £11 billion ($14 billion) in pharmaceutical R&D investment by 2033 unless urgent reforms are made to high and unpredictable clawback payments imposed on medicine sales to the National Health Service, according to a new report commissioned by the Association of the British Pharmaceutical Industry (ABPI). 3 June 2025
UK health technology assessor the National Institute for Health and Care Excellence (NICE) has published updated Final Draft Guidance recommending Forxiga (dapagliflozin) as an option to treat chronic kidney disease in adults, AstraZeneca announced today. 30 May 2025
The UK’s National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending that Filspari (sparsentan) can be used in the National Health Service (NHS) in England as an option to treat primary IgA nephropathy in adults with a urine protein excretion of 1.0g/day or more, or a urine protein-to-creatinine ratio of 0.75g/g or more. 23 May 2025
A week after US President Donald Trump signed an executive order to bring back the Most Favored Nation (MFN) idea he introduced at the end of his first term, the US Department of Health and Human Services (HHS announced it is proceeding with implementing the order on prescription drug pricing. 20 May 2025
A US district court judge has found that the Department of Health and Human Services (HHS) must preapprove the use of 340B rebate models before they can be implemented. 19 May 2025
The European Federation of Pharmaceutical Industries and Associations (EFPIA) has released new data that sheds light on why patients in some European countries wait significantly longer than others for access to innovative medicines. 19 May 2025
The Institute for Clinical and Economic Review (ICER) has released a Final Evidence Report assessing the comparative clinical effectiveness and value of sonpiretigene isteparvovec for the treatment of advanced retinitis pigmentosa. 16 May 2025
US President Donald Trump released an executive order on May 12 calling for a 30-day negotiation period between the Department of Health and Human Services (HHS) and the biopharma industry, with the threat of a rule from the Centers for Medicare and Medicaid Services (CMS) to lower US drug prices if no deal is reached. 14 May 2025
US pharma stocks regained ground Monday after Donald Trump signed an executive order aiming to slash prescription drug prices, a move that initially rattled investors but left many analysts unconvinced about its impact. 13 May 2025
US President Donald Trump sent shivers through the pharmaceutical industry as he vowed to dramatically reduce the high drug prices that have long been a feature of the US market. 12 May 2025
New figures from European industry group EFPIA paint a stark picture of the growing divide in access to innovative medicines across the EU, with patients in some countries waiting more than seven times longer than others for the same treatment. 12 May 2025
UK pharma major AstraZeneca has suffered a setback in its legal fight to block the US government's efforts to lower prescription drug prices, after a federal appeals court dismissed its challenge to Medicare’s negotiation powers. 9 May 2025
The introduction of outpatient flat-rate payments approved today by the Swiss Federal Council poses significant risks for patients and for the pharmaceutical industry as a whole. Including medication costs in flat-rate payments creates a dangerous perverse incentive to treat patients with the cheapest rather than the most suitable medications, according to a website posting by Interpharma. 5 May 2025